CN109952114A - 用于治疗醛脱氢酶缺乏的基因疗法 - Google Patents

用于治疗醛脱氢酶缺乏的基因疗法 Download PDF

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Publication number
CN109952114A
CN109952114A CN201780058722.4A CN201780058722A CN109952114A CN 109952114 A CN109952114 A CN 109952114A CN 201780058722 A CN201780058722 A CN 201780058722A CN 109952114 A CN109952114 A CN 109952114A
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China
Prior art keywords
carrier
aldehyde dehydrogenase
mammal
aldh2
ala
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CN201780058722.4A
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English (en)
Chinese (zh)
Inventor
M·加斯米
R·G·克雷斯托
O·E·帕格维齐
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Cornell University
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Cornell University
Adverum Biotechnologies Inc
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Publication of CN109952114A publication Critical patent/CN109952114A/zh
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/09—Recombinant DNA-technology
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    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
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    • C12Y101/01001—Alcohol dehydrogenase (1.1.1.1)
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN201780058722.4A 2016-07-26 2017-07-26 用于治疗醛脱氢酶缺乏的基因疗法 Pending CN109952114A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662367012P 2016-07-26 2016-07-26
US62/367,012 2016-07-26
PCT/US2017/043999 WO2018022783A1 (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency

Publications (1)

Publication Number Publication Date
CN109952114A true CN109952114A (zh) 2019-06-28

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Application Number Title Priority Date Filing Date
CN201780058722.4A Pending CN109952114A (zh) 2016-07-26 2017-07-26 用于治疗醛脱氢酶缺乏的基因疗法

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Country Link
US (1) US20190160187A1 (https=)
EP (1) EP3490613A4 (https=)
JP (2) JP7165357B2 (https=)
KR (1) KR102621134B1 (https=)
CN (1) CN109952114A (https=)
AU (1) AU2017301819B2 (https=)
SG (2) SG10202100632WA (https=)
WO (1) WO2018022783A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114891808A (zh) * 2022-06-21 2022-08-12 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物

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US20220226502A1 (en) * 2019-06-03 2022-07-21 Institute For Cancer Research d/b/aThe Research Institute of Fox Chase Cancer Center Adeno-associated virus vector delivery of cystathionine beta-synthase (cbs) enzyme for treating cbs deficiency
WO2021119142A1 (en) * 2019-12-09 2021-06-17 The Trustees Of Columbia University In The City Of New York 3d organoids for personalized oral cancer therapy
CN111471660B (zh) * 2020-03-12 2023-11-24 广州辉园苑医药科技有限公司 一种乙醛脱氢酶重组基因及其乳酸菌载体和应用
KR102557375B1 (ko) * 2020-12-16 2023-07-20 동국대학교 와이즈캠퍼스 산학협력단 미토콘드리아 전구서열 유래 펩타이드 및 그 용도
GB202218090D0 (en) * 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

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US20130267501A1 (en) * 2008-10-28 2013-10-10 The Board Of Trustees Of The Leland Stanford Junior University Modulators of Aldehyde Dehydrogenase and Methods of Use Thereof
US20140221462A1 (en) * 2013-02-01 2014-08-07 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

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CA2530248A1 (en) * 2003-06-25 2005-01-06 Gencia Corporation Modified vectors for organelle transfection
WO2009137045A2 (en) * 2008-05-07 2009-11-12 The Board Of Trustees Of The Leland Stanford Junior University Crystal structure of aldehyde dehydrogenase and methods of use thereof
EP2561073B1 (en) * 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
AU2014257131B9 (en) * 2013-04-23 2019-12-05 Nizyme, Inc. Methods and compositions for treating diseases
HUE051255T2 (hu) * 2014-02-19 2021-03-01 Aviv Therapeutics Inc Mitokondriális aldehid dehidrogenáz 2 (ALDH2) kötõ policiklusos amidok és alkalmazásuk rák kezelésére
CA3008264A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
MX2018007234A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.

Patent Citations (2)

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US20130267501A1 (en) * 2008-10-28 2013-10-10 The Board Of Trustees Of The Leland Stanford Junior University Modulators of Aldehyde Dehydrogenase and Methods of Use Thereof
US20140221462A1 (en) * 2013-02-01 2014-08-07 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

Non-Patent Citations (4)

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Title
FRANC¸ OISE PIGUET，等: "Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice", 《HUMAN GENE THERAPY》 *
STEFANIE HAUG Æ MARKUS BRAUN-FALCO: "Adeno-associated virus vectors are able to restore fatty aldehyde dehydrogenase-deficiency. Implications for gene therapy in Sjo¨ gren-Larsson syndrome", 《ARCH DERMATOL RES》 *
X.Y. HU，等: "Aldehyde dehydrogenase 2 protects human umbilical vein endothelial cells against oxidative damage and increases endothelial nitric oxide production to reverse nitroglycerin tolerance", 《GENETICS AND MOLECULAR RESEARCH》 *
YUKI MATSUMURA，等: "Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114891808A (zh) * 2022-06-21 2022-08-12 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物
CN114891808B (zh) * 2022-06-21 2023-10-27 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物

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KR102621134B1 (ko) 2024-01-04
KR20190038583A (ko) 2019-04-08
EP3490613A4 (en) 2020-03-04
AU2017301819A1 (en) 2019-02-07
SG11201900543SA (en) 2019-02-27
EP3490613A1 (en) 2019-06-05
JP7165357B2 (ja) 2022-11-04
WO2018022783A1 (en) 2018-02-01
US20190160187A1 (en) 2019-05-30
SG10202100632WA (en) 2021-03-30
JP2019521692A (ja) 2019-08-08
AU2017301819B2 (en) 2024-09-26
JP2022116045A (ja) 2022-08-09
JP7558535B2 (ja) 2024-10-01

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